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FI3303598T3 - Targeted lipid particles for systemic delivery of nucleic acid molecules to leukocytes - Google Patents

Targeted lipid particles for systemic delivery of nucleic acid molecules to leukocytes Download PDF

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Publication number
FI3303598T3
FI3303598T3 FIEP16799476.3T FI16799476T FI3303598T3 FI 3303598 T3 FI3303598 T3 FI 3303598T3 FI 16799476 T FI16799476 T FI 16799476T FI 3303598 T3 FI3303598 T3 FI 3303598T3
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Finland
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peg
particle
glycero
leukocyte
lymphoma
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FIEP16799476.3T
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Finnish (fi)
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Dan Peer
Shiri Weinstein
Itai Antoine Toker
Srinivas Ramishetti
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Univ Ramot
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/095—Sulfur, selenium, or tellurium compounds, e.g. thiols
    • A61K31/105—Persulfides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6849—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C07K16/2812—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily against CD4
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2896—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against molecules with a "CD"-designation, not provided for elsewhere

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Claims (15)

PATENTTIVAATIMUKSETPATENT CLAIMS 1. Nukleiinihappoa kapseloiva partikkeli nukleiinihapon annostelemiseksi kohdennettuun annosteluun leukosyyttisoluun, jolloin partikkeli käsittää: lipidiseosta, — joka sisältää kationista lipidiä, kalvoa stabiloivaa lipidiä ja kohdistusyksikköön konjugoitua PEG-maleimidiä; jolloin kohdistusyksikkö käsittää anti-CD38-vasta-aineen tai vasta-aineen, joka on valittu ryhmästä, jonka muodostavat anti-CD4-vasta-aine, anti- CDS8-vasta-aine ja anti-CD3-vasta-aine. 1. A nucleic acid encapsulating particle for delivering nucleic acid for targeted delivery to a leukocyte cell, the particle comprising: a lipid mixture, — comprising a cationic lipid, a membrane stabilizing lipid, and PEG-maleimide conjugated to a targeting moiety; wherein the targeting moiety comprises an anti-CD38 antibody or an antibody selected from the group consisting of an anti-CD4 antibody, an anti-CD58 antibody, and an anti-CD3 antibody. 2 Patenttivaatimuksen 1 mukainen partikkeli, jolloin kohdistusosa on anti-CD38- vasta-aine.The particle of claim 1, wherein the targeting moiety is an anti-CD38 antibody. 3. Patenttivaatimuksen 1 mukainen partikkeli, jolloin leukosyyttisolut ovat primaarisia lymfosyyttejä, edullisesti jossa lymfosyytit — on valittu B-soluista ja T-soluista; tai jolloin kohdistusosa on konfiguroitu erityisesti kohdistumaan leukosyyttiin; tai jolloin kohdistusosa on konfiguroitu tunnistamaan spesifisesti mainitun leukosyytin ekspressoima antigeeni. 3. The particle of claim 1, wherein the leukocyte cells are primary lymphocytes, preferably wherein the lymphocytes — are selected from B cells and T cells; or wherein the targeting moiety is configured to specifically target a leukocyte; or wherein the targeting moiety is configured to specifically recognize an antigen expressed by said leukocyte. 4 Patenttivaatimuksen 1 mukainen partikkeli, jonka leukosyyttisolu sisäänsä.4. A particle according to claim 1, having a leukocyte cell therein. 5. —Patenttivaatimuksen 4 mukainen partikkeli, jolloin leukosyyttisolu internalisoi partikkelin 30-120 minuutin kuluessa partikkelien systeemisestä antamisesta.5. —The particle of claim 4, wherein the particle is internalized by a leukocyte cell within 30-120 minutes after systemic administration of the particles. 6. —Patenttivaatimuksen 1 mukainen partikkeli, jolloin nukleiinihappoon sisältyy häiritsevä RNA, joka on valittu seuraavista: siRNA, miRNA, shRNA ja antisense-RNA, niiden muunnetut muodot tai niiden yhdistelmät: tai jolloin nukleiinihapon kapseloitumisprosentti on yli noin 90 %.6. —The particle of claim 1, wherein the nucleic acid comprises an interfering RNA selected from: siRNA, miRNA, shRNA, and antisense RNA, modified forms thereof, or combinations thereof: or wherein the percentage of nucleic acid encapsulation is greater than about 90%. 7. — Patenttivaatimuksen 1 mukainen partikkeli,7. — A particle according to claim 1, jolloin kationinen lipidi on valittu seuraavista: DLinDMA, DLin-MC3-DMA, DLin-KC2-DMA, di-oleyyli-sukkinyyli-seryyli-tobramysiini, di-oleyyli-adipyyli- tobramysiini, di-oleyyli-suberyyli-tobramysiini, N,N-dimetyyli-N' N'-di[(9Z, 122)- oktadeka-9,12-dien-1-yyli] etaani-1,2-diamiini, di-oleyyli-sebasyyli-tobramysiini, di-wherein the cationic lipid is selected from: DLinDMA, DLin-MC3-DMA, DLin-KC2-DMA, di-oleyl-succinyl-seryl-tobramycin, di-oleyl-adipyl-tobramycin, di-oleyl-suberyl-tobramycin, N,N-dimethyl-N' N'-di[(9Z, 122)-octadeca-9,12-dien-1-yl]ethane-1,2-diamine, di-oleyl-sebacyl-tobramycin, di- — oleyyli-ditioglykolyyli-tobramysiini, monokationinen lipidi N-[1-(2,3-dioleoyylioksi)]- N,N,N-trimetyyliammoniumpropaani (DOTAP), BCAT O-(2R-1,2-di-O-(1'Z, 9'Z- oktadekadienyyli)-glyseroli)-3-N-(bis-2-aminoetyyli)-karbamaatti, BGSC (bis- guanidinium-spermidiini-kolesteroli), BGTC (bis-guanidinium-tren-kolesteroli), CDAN (N'-kolesteryylioksikarbonyyli-1-3,7-diatsanonaani-1,9-diamiini), CHDTAEA— oleyl-dithioglycolyl-tobramycin, monocationic lipid N-[1-(2,3-dioleoyloxy)]-N,N,N-trimethylammoniumpropane (DOTAP), BCAT O-(2R-1,2-di-O-(1'Z,9'Z- octadecadienyl)-glycerol)-3-N-(bis-2-aminoethyl)carbamate, BGSC (bis- guanidinium-spermidine-cholesterol), BGTC (bis-guanidinium-tren-cholesterol), CDAN (N'-cholesteryloxycarbonyl-1-3,7-diazanonan-1,9-diamine), CHDTAEA (kolesteryylihemiditioglykolyylitris(amino(etyyli)amiini), DCAT (O-(1,2-di-O-(9'Z- oktadekanyyli)-glyseroli)-3-N-(bis-2-aminoetyyli)-karbamaatti), DC-kolesteroli (3p [N- (N', N'-dimetyyliaminoetaani)-karbamoyyli] kolesteroli), DLKD (O,O'-dilauryyli-N- lysyyliaspartaatti), DMKD (O,O'-dimyristyyli-N-lysyyliaspartaatti), DOG (diolsyyliglyseroli, DOGS (dioktadekyyliamidoglysyylispermiini), DOGSDSO (1,2-(cholesterylhemidithioglycolyltris(amino(ethyl)amine), DCAT (O-(1,2-di-O-(9'Z-octadecanyl)-glycerol)-3-N-(bis-2-aminoethyl)-carbamate), DC-cholesterol (3p [N-(N', N'-dimethylaminoethane)-carbamoyl] cholesterol), DLKD (O,O'-dilauryl-N-lysyl aspartate), DMKD (O,O'-dimyristyl-N-lysyl aspartate), DOG (diolsylglycerol, DOGS (dioctadecylamidoglycylspermine), DOGSDSO (1,2- — dioleoyyli-sn-glysero-3-sukkinyyli-2-hydroksietyylidisulfidiornitiini), DOPC (1,2- dioleoyyli-sn-glysero-3-fosfokoliini), DOPE (1,2-dioleoyyli-sn-glyseroli-3- fosfoetanoliamiini), DOSN (dioleyylisukkinyylietyylitioneomysiini), DOSP (dioleyylisukkinyyliparomysiini), DOST (dioleyylisukkinyylitobramysiini), 1,2-uikoyyli- 3-trimetyyliammoniopropaani, DOTMA (N'[1-(2,3-dioleyylioksi)propyyli]-N,N,N-— dioleoyl-sn-glycero-3-succinyl-2-hydroxyethyldisulfideornithine), DOPC (1,2-dioleoyl-sn-glycero-3-phosphocholine), DOPE (1,2-dioleoyl-sn-glycerol-3-phosphoethanolamine), DOSN (dioleoylsuccinylethylthioneomycin), DOSP (dioleoylsuccinylparomycin), DOST (dioleoylsuccinyltobramycin), 1,2-uicoyl-3-trimethylammoniopropane, DOTMA (N'[1-(2,3-dioleoyloxy)propyl]-N,N,N- — trimetyyliammoniumkloridi), DPPES (dipalmitoyylifosfatidyylietanolamidospermiini), DDAB ja DODAP tai niiden yhdistelmä; tai jolloin kalvoa stabiloiva lipidi on valittu ryhmästä, joka koostuu seuraavista kolesteroli, fosfolipidit, kefaliinit, sfingolipidit ja glykoglyserolipidit; tai joka käsittää lisäksi fosfatidyyliamiinia, joka on valittu seuraavista: 1,2-dilauroyyli-— trimethylammonium chloride), DPPES (dipalmitoylphosphatidylethanolamidospermine), DDAB and DODAP or a combination thereof; or wherein the membrane stabilizing lipid is selected from the group consisting of cholesterol, phospholipids, cephalins, sphingolipids and glycoglycerolipids; or which further comprises a phosphatidylamine selected from: 1,2-dilauroyl- —L-fosfatidyylietanoliamiini (DLPE), 1,2-dioleoyyli-sn-glysero-3-fosfoetanoliamiini (DOPE), 1,2-difytanoyyli-sn-glysero-3-fosfoetanoliamiini (DPhPE), 1,3-dipalmitoyyli- sn-glysero-2-fosfoetanoliamiini (1,3-DPPE), 1-palmitoyyli-3-oleoyyli-sn-glysero-2- fosfoetanoliamiini (1,3-POPE), biotiini-fosfatidyylietanoliamiini, 1,2-dimyristoyyli-sn- glysero-3-fosfoetanoliamiini (DMPE), 1,2-distearoyyli-sn-glysero-3-fosfoetanoliamiini—L-phosphatidylethanolamine (DLPE), 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE), 1,2-diphytanoyl-sn-glycero-3-phosphoethanolamine (DPhPE), 1,3-dipalmitoyl- sn-glycero-2-phosphoethanolamine (1,3-DPPE), 1-palmitoyl-3-oleoyl-sn-glycero-2-phosphoethanolamine (1,3-POPE), biotin-phosphatidylethanolamine, 1,2-dimyristoyl-sn-glycero-3-phosphoethanolamine (DMPE), 1,2-distearoyl-sn-glycero-3-phosphoethanolamine (DSPE) ja dipalmitoyylifosfatidyylietanoliamiini (DPPE); tai jolloin lipidiseos käsittää kationisen lipidin, joka on valittu seuraavista: DLin-MC3- DMA, DLinDMA ja DLin-KC2-DMA:; kolesteroli; DSPC; PEG-DMG: ja DSPE-PEG- maleimidi.(DSPE) and dipalmitoylphosphatidylethanolamine (DPPE); or wherein the lipid mixture comprises a cationic lipid selected from: DLin-MC3-DMA, DLinDMA and DLin-KC2-DMA; cholesterol; DSPC; PEG-DMG and DSPE-PEG-maleimide. 8. Patenttivaatimuksen 1 mukainen partikkeli käsittäen lisäksi yhden tai useamman PEG-johdannaisen; jolloin lisä-PEG-johdannainen on edullisesti valittu seuraavista: DMG-PEG, PEG- cDMA, 3-N-(-metoksipoly(etyleeniglykoli)-2000)karbamoyyli-1,2- dimyristyylioksipropyyliamiini; PEG-cDSA, 3-N-(-metoksipoly(etyleeniglykoli)- 2000)karbamoyyli-1,2-distearyylioksipropyyliamiini, PEG-amiini, DSPE-PEG tai niiden yhdistelmät. 8. The particle of claim 1, further comprising one or more PEG derivatives; wherein the additional PEG derivative is preferably selected from the following: DMG-PEG, PEG-cDMA, 3-N-(-methoxypoly(ethylene glycol)-2000)carbamoyl-1,2-dimyristyloxypropylamine; PEG-cDSA, 3-N-(-methoxypoly(ethylene glycol)-2000)carbamoyl-1,2-distearyloxypropylamine, PEG-amine, DSPE-PEG or combinations thereof. 9 Patenttivaatimuksen 1 mukainen koostumus käsittäen useita partikkeleita.9 The composition of claim 1 comprising a plurality of particles. 10. Farmaseuttinen koostumus käsittäen patenttivaatimuksen 9 mukaista koostumusta annosmuodossa, joka soveltuu annettavaksi oraalisen ja parenteraalisen antoreitin kautta, tai jolloin anto on systeemistä.A pharmaceutical composition comprising the composition of claim 9 in a dosage form suitable for administration via oral and parenteral routes of administration, or wherein the administration is systemic. 11. Patenttivaatimuksen 10 mukainen farmaseuttinen koostumus käytettäväksi leukosyyttiin liittyvän sairauden hoidossa.A pharmaceutical composition according to claim 10 for use in the treatment of a leukocyte-related disease. 12. Patenttivaatimuksen 11 mukaisesti käytettävä farmaseuttinen koostumus, — jolloin nukleiinihappo on siRNA, jolloin edullisesti siRNA on suunnattu solusyklin säätelijää vastaan, jolloin edullisemmin solusyklin säätelijä on sykliini D1.12. A pharmaceutical composition for use according to claim 11, - wherein the nucleic acid is siRNA, wherein preferably the siRNA is directed against a cell cycle regulator, wherein more preferably the cell cycle regulator is cyclin D1. 13. Patenttivaatimuksen 11 mukainen farmaseuttinen koostumus käytettäväksi, jossa leukosyyttiin liittyvä sairaus on syöpä.The pharmaceutical composition for use according to claim 11, wherein the leukocyte-related disease is cancer. 14. Patenttivaatimuksen 13 mukainen farmaseuttinen koostumus, jolloin syöpä on verisyöpä, joka on valittu joukosta lymfooma, leukemia ja multippeli myelooma.The pharmaceutical composition of claim 13, wherein the cancer is a blood cancer selected from the group consisting of lymphoma, leukemia and multiple myeloma. 15. Patenttivaatimuksen 14 mukaisesti käytettävä farmaseuttinen koostumus, jolloin lymfooma on valittu seuraavista B-solulymfooma ja T-solulymfooma; tai jolloin B-solulymfooma on valittu seuraavista Hodgkinin lymfooma ja non- Hodgkinin lymfooma; tai jolloin lymfooma on manttelisolulymfooma (MCL).15. The pharmaceutical composition for use according to claim 14, wherein the lymphoma is selected from B-cell lymphoma and T-cell lymphoma; or wherein the B-cell lymphoma is selected from Hodgkin's lymphoma and non-Hodgkin's lymphoma; or wherein the lymphoma is mantle cell lymphoma (MCL).
FIEP16799476.3T 2015-05-26 2016-05-25 Targeted lipid particles for systemic delivery of nucleic acid molecules to leukocytes FI3303598T3 (en)

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US201562268526P 2015-12-17 2015-12-17
PCT/IL2016/050543 WO2016189532A1 (en) 2015-05-26 2016-05-25 Targeted lipid particles for systemic delivery of nucleic acid molecules to leukocytes

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Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019071029A1 (en) * 2017-10-04 2019-04-11 City Of Hope Prevention and treatment of gvhd and autoimmune diseases
TWI894123B (en) 2017-12-27 2025-08-21 日商武田藥品工業有限公司 Lipid nanoparticles containing nucleic acid and their uses
CA3106146A1 (en) * 2018-07-13 2020-01-16 Genmab A/S Variants of cd38 antibody and uses thereof
KR20210093232A (en) 2018-10-09 2021-07-27 더 유니버시티 오브 브리티시 콜롬비아 Compositions and systems and related methods comprising transfection competent vesicles free of organic solvent and detergent
MX2021004357A (en) * 2018-10-18 2021-05-31 Takeda Pharmaceuticals Co Method for activation/proliferation of t cells.
US20220249389A1 (en) 2019-07-12 2022-08-11 Oregon Health & Science University Immunotherapeutic constructs and methods of their use
CA3139714A1 (en) 2019-07-12 2021-01-21 Wassana Yantasee Therapeutic constructs for co-delivery of mitotic kinase inhibitor and immune checkpoint inhibitor
JP2023546067A (en) * 2020-10-13 2023-11-01 ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア In vivo targeting of T cells for mRNA therapeutics
WO2022081702A1 (en) * 2020-10-13 2022-04-21 The Trustees Of The University Of Pennsylvania In vivo targeting of cd4+-t cells for mrna therapeutics
PH12023500013A1 (en) 2020-12-04 2024-03-11 Tidal Therapeutics Inc Ionizable cationic lipids and lipi nanoparticles, and methods of synthesis and use thereof
US20240307309A1 (en) * 2020-12-22 2024-09-19 Cornell University Zwitterionic lipid nanoparticle compositions, and methods of use
CN113116819B (en) * 2021-04-24 2022-09-20 郑州大学 siRNA-loaded nano lipid hybrid micelle and preparation method and application thereof
CA3215963A1 (en) * 2021-05-28 2022-12-01 Yaoxin LIN Lipid compound and use thereof in delivery of nucleic acid
WO2023023055A1 (en) 2021-08-16 2023-02-23 Renagade Therapeutics Management Inc. Compositions and methods for optimizing tropism of delivery systems for rna
EP4402123A1 (en) 2021-09-14 2024-07-24 Renagade Therapeutics Management Inc. Cyclic lipids and methods of use thereof
EP4402121A1 (en) 2021-09-14 2024-07-24 Renagade Therapeutics Management Inc. Acyclic lipids and methods of use thereof
CN114457111B (en) * 2021-10-19 2024-03-15 吉优诺（上海）基因科技有限公司 Gene delivery system and preparation method and application thereof
WO2023081756A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
EP4452928A1 (en) 2021-12-23 2024-10-30 Renagade Therapeutics Management Inc. Constrained lipids and methods of use thereof
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
CA3255619A1 (en) 2022-04-07 2023-10-12 Renagade Therapeutics Man Inc Cyclic lipids and lipid nanoparticles (lnp) for the delivery of nucleic acids or peptides for use in vaccinating against infectious agents
WO2023240156A1 (en) 2022-06-08 2023-12-14 Tidal Therapeutics, Inc. Ionizable cationic lipids and lipid nanoparticles, and methods of synthesis and use thereof
KR20250065887A (en) * 2022-10-19 2025-05-13 후지필름 가부시키가이샤 Lipid compositions and methods of delivering therapeutic agents
WO2024182707A1 (en) 2023-03-02 2024-09-06 Krystal Biotech, Inc. Interleukin-2 and interleukin-12 for cancer therapy
WO2024190817A1 (en) * 2023-03-13 2024-09-19 富士フイルム株式会社 Compound or salt thereof, lipid composition, pharmaceutical composition, and delivery carrier
AU2024236558A1 (en) 2023-03-15 2025-10-09 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
WO2024192277A2 (en) 2023-03-15 2024-09-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
AR132276A1 (en) 2023-04-07 2025-06-11 Takeda Pharmaceuticals Co CONJUGATION COMPLEX
CN121079317A (en) * 2023-04-28 2025-12-05 深圳深信生物科技有限公司 Modified delivery vehicles and uses thereof
US12311033B2 (en) 2023-05-31 2025-05-27 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025076113A1 (en) 2023-10-05 2025-04-10 Capstan Therapeutics, Inc. Ionizable cationic lipids with conserved spacing and lipid nanoparticles
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025166238A1 (en) 2024-01-31 2025-08-07 Orna Therapeutics, Inc. Fast-shedding polyethylene glycol lipids
WO2025174825A2 (en) 2024-02-12 2025-08-21 Aera Therapeutics, Inc. Delivery compositions
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025217454A2 (en) 2024-04-11 2025-10-16 Capstan Therapeutics, Inc. Ionizable cationic lipids and lipid nanoparticles
WO2025217452A1 (en) 2024-04-11 2025-10-16 Capstan Therapeutics, Inc. Constrained ionizable cationic lipids and lipid nanoparticles

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110038941A1 (en) 2007-12-27 2011-02-17 The Ohio State University Research Foundation Lipid Nanoparticle Compositions and Methods of Making and Using the Same
US9018187B2 (en) * 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
EP2580243B1 (en) 2010-06-09 2019-10-16 Genmab A/S Antibodies against human cd38
KR20140102759A (en) 2011-12-16 2014-08-22 모더나 세라퓨틱스, 인코포레이티드 Modified nucleoside, nucleotide, and nucleic acid compositions
JP2015509085A (en) * 2012-01-01 2015-03-26 キュービーアイ エンタープライゼズ リミテッドＱｂｉ Ｅｎｔｅｒｐｒｉｓｅｓ Ｌｔｄ． Particles targeting ENDO180 for selective delivery of therapeutic and diagnostic agents
WO2015198326A1 (en) * 2014-06-26 2015-12-30 Ramot At Tel-Aviv University Ltd. Liposomal formulations for delivery of nucleic acids

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